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Saxtons River teen suffering from rare condition speaks in D.C.

By DOMENIC POLI / Reformer Staff

Posted:
02/16/2013 03:00:00 AM EST

Thirteen-year-old Austin and 10-year-old Max Leclaire play together with their Ben 10 action figures in Saxtons River home. The brothers both suffer from Duchenne muscular dystrophy, but because of a clinical trial drug, Eteplirsen, Max has retained his ability to walk while his brother's muscles continue to deteriorate. (Josh Stilts/Reformer file photo)

Saturday February 16, 2013

BRATTLEBORO -- Austin Leclaire's family was in Washington, D.C., on a mission Wednesday.

Austin and his mother, Jenn McNary, were in the nation's capital to deliver a petition with more than 170,000 signatures calling on the U.S. Food and Drug Administration to expedite the approval of Eteplirsen, a drug that could save Austin's life. The 14-year-old Saxtons River boy and his younger brother, Max, both suffer from Duchenne Muscular Dystrophy, an ultimately fatal degenerative muscle disease that affects roughly 20,000 children in the United States.

The boys' father, Craig McNary, and Max and were also in Washington.

Max, 11, was eligible for a medical trial of Eteplirsen, which greatly improved his quality of life. Austin, however, did not qualify and his condition has gotten progressively worse without the treatment. Max is able to walk while his older brother is confined to a wheelchair.

Their family was among dozens joined by U.S. Rep. Mike Fitzpatrick (R-Penn.) asking the FDA to accelerate its approval of the "miracle drug."

Austin and Jenn both spoke in Room 2325 of the Rayburn House Office Building to explain how Eteplirsen could improve the lives of people with DMD.

"It went really well. There wasn't a dry eye in the house," Jenn told the Reformer via cell phone while negotiating the D.C. Metrorail system Wednesday. "We didn't get a response from the FDA and not much press showed up, but I could not be prouder of our team.

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Craig and the boys flew home Thursday while Jenn remained in the capital to meet with Dr. Janet Woodcock, the FDA's director of the Center for Drug Evaluation and Research, on Friday.

Austin told the audience in the Rayburn House Office Building about how Eteplirsen has helped his brother, who can is now stronger and can run more. He said he is getting weaker without the drug.

"I enjoyed speaking," he told the Reformer. "I told them everything."

Sandy Walsh, with the FDA Office of Public Affairs, said the administration's staff has been in contact with the family and are discussing their interests. However, federal law prohibits anyone at the FDA from discussing any particular drug that may be under review or in clinical trials.

According to the FDA's website, investigational or experimental drugs are new drugs that have not yet been approved by the FDA or approved drugs that have not yet been approved for a new use, and are in the process of being tested for effectiveness and safety. These drugs "are available through two pathways designed to protect patients, because an investigational drug may pose unknown risks to patients and we do not know if it is effective."

Craig, Austin and Max met with U.S. Rep. Peter Welch (D-Vt.) in the congressman's Washington office after the press conference that followed the event at the Rayburn House Office Building. The three, accompanied by new friend Jake Wesley, a boy who also has DMD, chatted with Welch for about a half-hour and took a couple of photographs in his office.

Welch told the Reformer his guests made quite an impression on him.

"It was pretty moving to see parents full-on dedicated to helping their children get through this," he said, adding that he is throwing his support behind their cause. "The trial seemed to have a positive impact on the youngest boy and the hope is that (the drug) might be beneficial to others."

He admitted, however, that FDA approval is not a simple and clear-cut as it may seem.

"Whenever you're developing new medication, there needs to be a scientific and medical analysis (to prove) that it's safe," Welch said. "It's not something you can do by drawing up a diagram or an equation."

The congressman said Fitzpatrick, his Republican colleague from Pennsylvania, is becoming a good friend of his and is a fellow member of "No Labels," a group he said is comprised of 20 Democrats and 20 Republicans dedicated to working together to fix the country's problems regardless of party politics.

Fitzpatrick said too many young boys are diagnosed with Duchenne muscular dystrophy every year and the drug treatments currently available are not fully meeting the needs of families affected by it.

"There are several promising improvements in treatment making their way through the standard FDA approval process, but this progress is not helping those most in need. I have reached out to the FDA and urged them to consider placing these drugs into their Accelerated Approval Program," he said in a statement to the Reformer. "This program prioritizes the approval of the most effective drugs to provide greater relief to those diagnosed with serious illnesses like Duchenne muscular dystrophy. I remain hopeful that this expedited process will make these medical innovations accessible to the entire DMD community."

The family also meet with Sen. Patrick Leahy, D-Vt., who released a statement to the Reformer.

"I share the McNarys' belief that the FDA should ensure the safety and effectiveness of all drugs that are brought to the marketplace," Leahy stated. "I was also pleased to join with eight other senators this week to introduce the MD CARE Act. Since its original passage in 2001, that law has helped to develop important therapies for those suffering from MD and to extend life expectancy by 10 years."

Craig was glad to meet with Welch and Leahy after the press conference and learn his family has the congressman's support, but said it was a little disheartening not to have any of Vermont's representatives to the nation's capital actually at the speeches. Still, he feels his family has plenty of backing for their cause.

"I think we got a lot of support. It's a good beginning, but I think there's more work to do," he said. "Obviously, you don't get anything done just one time in Washington."

Jenn will revisit in late March and she and her husband expect to go again later in the year.

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